Adrenas Therapeutics, Inc.

Dr. Gao has made foundational contributions in the discovery and characterization of adeno-associated virus (AAV) serotypes which were instrumental in the resurgence of gene therapy GENEVA, SWITZERLAND / ACCESSWIRE / April 3, 2023 / RELIEF THERAPEUTICS Holding SA (SIX:RLF)(OTCQB:RLFTF)(OTCQB:RLFTY ) ("Relief Therapeutics" or the "Company"), a biopharmaceutical company committed to advancing treatment paradigms to benefit the lives of patients living with rare diseases, today announces the appointment of world-renowned gene therapy pioneer Guangping Gao, Ph.D. as the chair of the Company's newly formed scientific advisory board (SAB). Dr. Gao is the co-director of the Li Weibo Institute for Rare Diseases Research, director of the Horae Gene Therapy Center and Viral Vector Core, professor of microbiology and physiological systems, and Penelope Booth Rockwell Chair in biomedical research at the University of Massachusetts Chan Medical School in Worcester, Mass. Internationally recognized in the field of gene therapy, Dr. Gao played a key role in the discovery and characterization of a new family of adeno-associated virus (AAV) serotypes, which was instrumental in reviving the gene therapy field, significantly impacting many currently untreatable human diseases. "Dr. Gao is a distinguished and accomplished academic and genetic medicine leader who has made fundamental discoveries in gene therapy that have pushed the field forward. He brings an unparalleled combination of pioneering scientific research and pre-clinical and clinical gene therapy product development expertise, along with advanced viral vector manufacturing experience," said Serene Forte, Ph.D., MPH , senior vice president, head of genetic medicine at Relief Therapeutics. "It will be a privilege to work with Dr. Gao and benefit from his vast knowledge and advice in this space." In his advisory role, Dr. Gao will serve as an integral resource, providing scientific review and high-level technical and strategic guidance related to gene therapy targets, research and pre-clinical development and strategic research alliances as the Company works to expand its portfolio. "The Relief Therapeutics team has built a strong foundation with their current portfolio and cost-effective capital approach to drug development," said Dr. Gao. "I look forward to working with Dr. Forte and the highly capable leadership team to help build and proficiently execute the Relief Therapeutics genetic medicine program." Relief Therapeutics launched its genetic medicines initiative with the objective of developing life-altering, potentially curative therapies for patients suffering from devastating rare diseases that currently lack treatment options. The company is leveraging its strength and experience to identify monogenic disorders in therapeutic areas that align with its areas of focus, such as rare metabolic diseases. The SAB will support the Company's genetic medicines initiative and additional appointments are forthcoming. To learn more about Relief Therapeutics' approach to genetic medicine, please visit: . "Dr. Gao's extensive experience in the advancement of novel approaches to treat inherited rare diseases will be instrumental in helping us shape the direction of our genetic medicine portfolio," said Jack Weinstein , chief executive officer at Relief Therapeutics. "We are honored to have Dr. Gao lead our new SAB and welcome him to Relief Therapeutics." ABOUT GUANGPING GAO, PH.D. Guangping Gao, Ph.D. is co-director of the Li Weibo Institute for Rare Diseases Research, director of the Horae Gene Therapy Center and Viral Vector Core, professor of microbiology and physiological systems, and Penelope Booth Rockwell Chair in biomedical research at the University of Massachusetts Chan Medical School in Worcester, Mass. Dr. Gao is an elected fellow, both at the U.S. National Academy of Inventors (NAI) and American Academy of Microbiology. For more than 30 years, Dr. Gao has primarily focused on molecular genetics and viral vector gene therapy of rare genetic diseases, encompassing disease gene cloning, causative mutation identification, pathomechanism investigation, animal modeling, novel viral vector discovery and engineering for in vivo gene delivery, vector biology, pre-clinical and clinical gene therapy product development, viral vector manufacturing for pre-clinical and clinical gene therapy applications as well as technology platform development as novel approaches for human gene therapy. Dr. Gao has published nearly 350 research papers, six book chapters and five edited books. He holds 233 patents with 480 more patent applications pending. Dr. Gao serves as the executive editor-in-chief of Human Gene Therapy , senior editor of the Gene and Cell Therapy book series, associate editor of Signal Transduction and Targeted Therapy and serves on the editorial boards of several other gene therapy and virology journals. Nature Biotechnology has ranked Dr. Gao as one of the top 20 translational researchers in the world for several years in a row. From 2017 to 2020, he served as vice president, president-elect and president of the American Society of Gene and Cell Therapy. Dr. Gao is a co-founder of Voyager Therapeutics, Adrenas Therapeutics and Aspa Therapeutics, which are focused on developing recombinant adeno-associated virus (rAAV) gene therapeutics for treating a variety of devastating rare diseases. Dr. Gao earned a bachelor of medicine degree from West China School of Medicine, Sichuan University in Chengdu, China. He earned both a master of science degree and doctor of philosophy degree in biological sciences from Florida International University in Miami. Dr. Gao completed his postdoctoral training at the University of Pennsylvania Perelman School of Medicine in Philadelphia. ABOUT RELIEF THERAPEUTICS Relief Therapeutics is a commercial-stage biopharmaceutical company committed to advancing treatment paradigms and delivering improvements in efficacy, safety and convenience to benefit the lives of patients living with rare diseases. Since founding in 2013, Relief Therapeutics continues to build a diversified pipeline of risk-mitigated assets to address metabolic, dermatology/connective tissue disorders as well as pulmonary and genetic diseases. Our portfolio also includes a balanced mix of marketed, revenue-generating products and the proprietary, globally patented Physiomimic™ and Tehclo ® platform technologies which were obtained through the acquisition of APR Applied Pharma Research S.A. in June 2021. Our mission is being advanced by an international team of well-established, experienced biopharma industry leaders with extensive research, development and rare disease expertise. Relief Therapeutics' headquarters are located in Geneva, with additional offices in Balerna, Switzerland, Rome, Italy and Offenbach am Main, Germany. The Company is listed on the SIX Swiss Exchange under the symbol RLF and quoted in the U.S. on OTCQB under the symbols RLFTF and RLFTY. For more information, please visit or follow Relief Therapeutics on LinkedIn and Twitter . FOR MEDIA/INVESTOR INQUIRIES CONTACT: RELIEF THERAPEUTICS Holding SA Catherine Day Vice President, IR & Communications catherine.day@relieftherapeutics.com LifeSci Advisors Irina Koffler ikoffler@lifesciadvisors.com DISCLAIMER This communication expressly or implicitly contains certain forward-looking statements concerning RELIEF THERAPEUTICS Holding SA and its businesses. Such statements involve certain known and unknown risks, uncertainties and other factors, including those risks described in RELIEF THERAPEUTICS Holding SA's press releases and filings with the SIX Swiss Exchange and the U.S. Securities and Exchange Commission, all of which could cause the actual results, financial condition, performance or achievements of RELIEF THERAPEUTICS Holding SA to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. RELIEF THERAPEUTICS Holding SA is providing this communication as of this date and does not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise. SOURCE: Relief Therapeutics Holdings AG View source version on accesswire.com:

DUBLIN--( BUSINESS WIRE )--The "AAV vectors in gene therapy - Pipeline Insight, 2022" drug pipelines has been added to ResearchAndMarkets.com's offering. AAV vectors in gene therapy: Overview Out of the several viral vectors that have been used to date for delivering the genes of interest, the Adeno-associated viral (AAV) vector appears to be the safest and effective vehicle and can maintain long-term gene and protein expression following a single injection of the vector. AAV vectors are the leading viral vectors for gene delivery to treat a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs, and harnessing revolutionary biotechnologies have contributed extensively to the growth of the gene therapy field. AAV Vectors in Gene Therapy Emerging Drugs GS010: Gensight Biologics LUMEVOQ (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. "LUMEVOQ" was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018. Valoctocogene roxaparvovec: BioMarin Pharmaceutical Valoctocogene roxaparvovec is an investigational AAV5 gene therapy under regulatory review for the treatment of severe hemophilia A. It is currently in preregistration stage of development. In June 2021, BioMarin resubmitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA). In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase III GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration (FDA). BioMarin is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA. DTX401: Ultragenyx Pharmaceutical DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase-? under control of the native promoter. DTX401 is administered as a single intravenous infusion and has been shown in preclinical studies to improve G6Pase-? activity and reduce hepatic glycogen levels, a well-described biomarker of disease progression. In a Phase I/II clinical study, all nine patients showed a clinical response, with significant reductions in the need for cornstarch and improvements in glucose control and other metabolic parameters compared to baseline. The drug is currently being evaluated in Phase III clinical trial to treat patients with Glycogen storage disease type I. AAV5-RPGR: MeiraGTx AAV-RPGR is an investigational gene therapy for the treatment of patients with X-linked retinitis pigmentosa (XLRP) caused by disease-causing variants in the eye specific form of the RPGR gene (RPGR ORF15). AAV-RPGR is designed to deliver functional copies of the RPGR gene to the subretinal space in order to improve and preserve visual function. MeiraGTx and development partner Janssen are currently conducting a Phase III clinical trial of AAV-RPGR in patients with XLRP with disease-causing variants in RPGR ORF15. Timrepigene emparvovec: Biogen Timrepigene emparvovec is an AAV2 vector administered by subretinal injection, which aims to provide a functioning CHM gene and expression of the REP-1 protein to restore membrane trafficking and thereby slow, stop or potentially reverse decline in vision. Data from the Phase 1/2 studies demonstrated a slower rate of decline in visual acuity in patients treated with timrepigene emparvovec compared to untreated patients in the natural history study. In addition, some patients treated with timrepigene emparvovec showed improvements in visual acuity. The studies also demonstrated that timrepigene emparvovec was generally well tolerated with an acceptable safety profile. The safety and efficacy of a single subretinal injection of timrepigene emparvovec is currently being evaluated in the ongoing Phase III STAR study. AMT 061: UniQure AMT-061 (etranacogene dezaparvovec) is an experimental gene therapy that uniQure is developing to treat hemophilia B. AMT-061 uses a modified and harmless adeno-associated virus 5 (AAV5) to deliver a highly functional copy of the F9 gene, called FIX-Padua, to patients' cells. The FIX-Padua gene version was shown to result in FIX clotting activity eight times greater than that associated with the standard F9 gene. As such, the one-time therapy - administrated directly into the bloodstream - is expected to increase FIX levels, helping to prevent and control bleeds. If approved, the therapy will be marketed globally by CSL Behring, which closed a commercialization and licensing agreement with uniQure in May 2021. UniQure and CSL Behring expect to file a regulatory application to the U.S. Food and Drug Administration (FDA) in early 2022 seeking AMT-061's approval for hemophilia B. Research and Development. RGX-314: REGENXBIO RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina. REGENXBIO is advancing research in two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye. According to the company's pipeline, the drug is currently in the Phase III stage of development. SPK-8011: Spark Therapeutics Investigational SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the AAV-LK03 capsid, also referred to as Spark200, contains a codon-optimized human factor VIII gene under the control of a liver-specific promoter. The Food and Drug Administration (FDA) granted orphan-disease designation and breakthrough therapy designation in the US, while the European Commission has granted orphan designation to SPK-8011. NFS-01: Neurophth Investigational NR082, a novel recombinant adeno-associated viral serotype 2 vector (rAAV2) containing a codon-optimized of NADH-dehydrogenase subunit 4 (ND4) gene under the control of the cytomegalovirus promoter and enhancer, is a novel ophthalmic injection that is being developed for the treatment of Leber hereditary optic neuropathy (LHON) associated with ND4 mutations. It is currently being evaluated in Phase II/III clinical trial. GT 005: Gyroscope Therapeutics GT005 is designed as an AAV2-based one-time investigational gene therapy for GA secondary to AMD that is delivered under the retina. GT005 aims to restore balance to an overactive complement system, a part of the immune system, by increasing production of the CFI protein. Complement overactivation has been strongly correlated with the development and progression of AMD. The CFI protein regulates the activity of the complement system. It is believed that increasing CFI production could dampen the system's overactivity and reduce inflammation, with the goal of preserving a person's eyesight. Gyroscope is also evaluating GT005 in two Phase II clinical trials. Further product details are provided in the report AAV vectors in gene therapy: Therapeutic Assessment This segment of the report provides insights about the different AAV vectors in gene therapy drugs segregated based on following parameters that define the scope of the report, such as: Major Players in AAV vectors in gene therapy There are approx. 70+ key companies which are developing the therapies for AAV vectors in gene therapy. The companies which have their AAV vectors in gene therapy drug candidates in the most advanced stage, i.e. Preregistration include, BioMarin Pharmaceutical. Phases The publisher's report covers around 235+ products under different phases of clinical development like Late stage products (Phase III) Mid-stage products (Phase II) Early-stage product (Phase I) along with the details of Pre-clinical and Discovery stage candidates Discontinued & Inactive candidates Route of Administration Key Players BioMarin Pharmaceutical Gensight Biologics PTC therapeutics Ultragenyx Pharmaceutical MeiraGTx Pfizer Biogen uniQure Pfizer Ultragenyx Pharmaceutical REGENXBIO Biogen Spark therapeuics (Roche) Sarepta therapeutics Neurophth Therapeutics LYSOGENE Gyroscope Therapeutics Nanoscope Therapeutics Homology medicines Ultragenyx Pharmaceutical Passage Bio Freeline therapeutics Astellas Pharma Aspa therapeutics Adrenas Therapeutics ESTEVE Sio Gene Therapies Amicus therapeutics 4D Molecular therapeutics Taysha Gene Therapies Atsena Therapeutics BioMarin Pharmaceutical Abeona Therapeutics REGENXBIO uniQure Taysha Gene Therapies Asklepios BioPharmaceutical Sarepta Therapeutics Abeona Therapeutics Forge Biologics Sangamo therapeuics Bayer LogicBio therapeutics Solid Biosciences Applied Genetic Technologies Spark therapeuics (Roche) Eli lilly and company Lexeo Therapeutics Spark therapeutics Gensight Biologics Alcyone Lifesciences MeiraGTx Audentes therapeutics Vivet Therapeutics Takeda Astellas Gene Therapies Homology Medicines Adverum Biotechnologies Libella Gene Therapeutics Rocket Pharmaceuticals Brain Neurotherapy Bio, Inc. Ocugen Asklepios BioPharmaceutical Neurogene Jaguar Gene Therapy, LLC Axovia Therapeutics Coave therapeutics Novartis/ Arctos medical Novartis/Vedere Bio REGENXBIO Inc. StrideBio Abeona therapeutics Sanofi/ Sirion therapeutics Capsida Biotherapeutics Kriya therapeutics Neurophth Poseida Therapeutics, BridgeBio Pharma DiNAQOR/ Biomarin ViGeneron AskBio/Selecta Biosciences Prevail therapeutics Intas Pharmaceuticals Tenaya Therapeutics Scout Bio therapeutics Ocugen Oyster Point pharmaceuticals Cyprium Therapeutics Solid Biosciences Amicus Therapeutics Coave therapeutics AvantiBio Voyager Therapeutics/ Pfizer Aruvant Sciences Alcyone Lifesciences Gene Therapy Research Institution Co., Ltd. For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/ylu69s .